The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Subcutaneous (sc) injection of Cemdisiran
Clinical Trial Site
Sarajevo, Bosnia and Herzegovina
Clinical Trial Site
Calgary, Canada
Clinical Trial Site
Tallinn, Estonia
Clinical Trial Site
Tartu, Estonia
The effect of Cemdisiran on platelet count
Time frame: Week 32
The effect of Cemdisiran on hematological response as measured by platelet count
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH)
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on hematological response as measured by rescue plasma therapy
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on LDH response as measured by LDH
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on LDH response as measured by rescue plasma therapy
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy
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Clinical Trial Site
Tbilisi, Georgia
Clinical Trial Site
Riga, Latvia
Clinical Trial Site
Kaunas, Lithuania
Clinical Trial Site
Vilnius, Lithuania
Clinical Trial Site
Chisinau, Moldova
Clinical Trial Site
Skopje, North Macedonia
...and 2 more locations
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on serum creatinine levels
Time frame: up to 84 weeks
The effect of Cemdisiran on estimated glomerular filtration rate (eGFR)
Time frame: up to 84 weeks
The effect of Cemdisiran on adverse events (AEs)
Time frame: up to 108 weeks